Absstract of: AU2023376971A1
The present invention relates to peptides, proteins, nucleic acids and cells for use in immunotherapeutic methods. In particular, the present invention relates to the immunotherapy of cancer, in particular of hematological neoplasms, such as acute myeloid leukemia (AML). The present invention furthermore relates to tumor-associated T-cell peptide epitopes that can for example serve as active pharmaceutical ingredients of vaccine compositions that stimulate anti-tumor immune responses, or to stimulate T cells ex vivo and transfer into patients. Peptides bound to molecules of the major histocompatibility complex (MHC), or peptides as such, can also be targets of antibodies, soluble T-cell receptors, and other binding molecules.
Absstract of: US2025161279A1
The present disclosure provides methods of treating multiple myeloma in a subject with a Bcl-2 inhibitor, in particularly 2-((1H-pyrrolo 2,3-bpyridin-5-yl)oxy)-N-((4-((((1r,4r)-4-hydroxy-4-methylcyclohexyl)methyl)amino)-3-nitrophenyl)sulfonyl)-4-(2-((S)-2-(2-isopropylphenyl)pyrrolidin-1-yl)-7-azaspiro3.5nonan-7-yl)benzamide or a pharmaceutically acceptable salt thereof, or in combination with dexamethasone.
Absstract of: US2025161248A1
A method for treating chronic myelomonocytic leukemia (CMML), or for maintaining or enhancing the proliferation of wildtype hematopoietic stem cells in patients suffering from CMML, by administering to a patient in need thereof a pharmaceutical composition containing an effective amount of an inhibitor of the CXCL8 receptor.
Absstract of: US2025163096A1
The present invention relates to chemical compounds, the compounds for use in a method of treatment, particularly in a method of prophylaxis or treatment for cancer, a process for preparation of the compounds and pharmaceutical compositions comprising the compounds. The compounds may, in particular, be useful in the treatment of leukaemia, lymphoma and/or solid tumours in Homo sapiens. The compounds are derivatives of cordycepin (3′-deoxyadenosine).
Absstract of: US2025163066A1
The present invention is directed to the compounds of Formula (I)—PROTACs of MALT1. The PROTACs described herein can be useful in the treatment of diseases or disorders associated with MALT1, such as lymphoma. In particular, the invention is concerned with compounds and pharmaceutical compositions degrade MALT1, methods of treating diseases or disorders associated with MALT1, and methods of synthesizing these compounds.
Absstract of: US2025163174A1
The invention provides antibodies which bind to LY75. Nucleic acid molecules encoding the antibodies, expression vectors, host cells and methods for expressing the antibodies are also provided. The antibodies may be used for the treatment of cancer, including pancreatic cancer, ovarian cancer, breast cancer, colorectal cancer, esophageal cancer, skin cancer, thyroid cancer, lung cancer, multiple myeloma and lymphoma.
Absstract of: US2025163146A1
Graft-versus-host disease (GVHD) prophylaxis often consists of calcineurin inhibitor-based combinations that indiscriminately curtail T cell receptor signal transduction. This broad inactivation consequently impairs the function of alloreactive pathogenic T cells as well as beneficial regulatory T cells (Treg) and anti-tumor cytotoxic T lymphocytes (CTL). Due to this non-selective approach, GVHD prevention is incomplete and the graft-versus-leukemia (GVL) effect is jeopardized. Disclosed are isolated binding molecules that disrupt the interaction of DLL4 and Notchl and methods of their use, including, but not limited to the treatment of graft versus host disease.
Absstract of: US2025163172A1
Disclosed herein is a method of treating cancer, such as multiple myeloma, involving the combination of an anti-BCMA antigen binding protein (e.g., an anti-BCMA antibody) and a proteasome inhibitor (e.g. bortezomib). The combinations can also include an anti-inflammatory compound (e.g. dexamethasone).
Absstract of: US2025161412A1
Compositions and methods are provided to treat and prevent cancers, such as myelomas, and include adoptive cell therapies in combination with an IL-15 superagonist and one or more chemotherapeutic agents.
Absstract of: US2025163388A1
Disclosed herein are methods of deriving, producing, maintaining, or expanding embryonic stem cells (ESCs) from avian species and a culture medium used for such methods. According to various embodiments, the method includes culturing an embryo extracted from an avian egg in a culture medium to harvest cells from yolk of the avian egg; dissociating cells from the cultured embryo; isolating a morphologically undifferentiated ESC colony from the dissociated cells in a culture medium; and culturing the isolated ESC colony in the presence of ovotransferrin, thereby deriving ESCs. According to various embodiments, the culture medium includes a Wnt inhibitor; a protein kinase C (PKC) inhibitor; ovotransferrin; an inhibitor of activin receptor-like kinases-4, -5, and -7; and a leukemia inhibitory factor (LIF).
Absstract of: WO2025104669A1
The disclosure provides methods of treating a refractory or relapsed acute myeloid leukemia (AML) in a subject comprising administration of a cell comprising a nucleic acid molecule encoding a chimeric antigen receptor (CAR), wherein the CAR comprises an antibody or antigen-binding fragment thereof that binds IL-1RAP, a transmembrane domain, and an intracellular signaling domain comprising at least a stimulatory domain, wherein prior to administering the cell, the subject is preconditioned with a lymphodepleting chemotherapy (LDC).
Absstract of: WO2025106474A1
The invention provides therapeutic and diagnostic methods for the treatment of cancer, such as multiple myeloma (MM), with anti-fragment crystallizable receptor-like 5 (FcRH5)/anti-cluster of differentiation 3 (CD3) bispecific antibodies.
Absstract of: WO2025104184A1
The present invention provides an in vitro method of generating a haematopoietic cell, the method comprising: a) Providing a cell genetically modified to comprise a nucleotide sequence(s) encoding exogenous transcription factors, the exogenous transcription factors comprising an ETS family transcription factor, T-cell acute lymphocytic leukaemia protein 1 (Tal1) and a GATA family transcription factor, wherein expression of the exogenous transcription factors from the nucleotide sequence(s) is inducible by culture with an inducer, and wherein the cell comprises a detectable expression level of the exogenous transcription factors; and b) Culturing the genetically modified cell in a differentiation medium which does not comprise the inducer, such that the expression level of the exogenous transcription factors in the cell is reduced to a level whereby the cell differentiates into a haematopoietic cell. Also provided are genetically modified haematopoietic cells, genetically modified cells and therapeutic uses thereof.
Absstract of: WO2025104045A1
The present invention relates to a method of treating an anaplastic lymphoma kinase (ALK) fusion-positive solid tumour or central nervous system tumour, comprising administering to a subject in need of such treatment a therapeutically effective amount of alectinib, or a pharmaceutically acceptable salt thereof, wherein the subject is aged <18 years.
Absstract of: WO2025106732A1
Single-nucleotide variants (SNVs) in key T cell genes can drive clinical pathologies and could be repurposed to improve cellular cancer immunotherapies. Here, we perform massively parallel base editing screens to generate thousands of variants at gene loci annotated with known or potential clinical significance. We discover a broad landscape of putative gain- (GOF) and loss-of-function (LOF) mutations, including in PIK3CD and its regulatory subunit PIK3R1, LCK, SOS, AKT1, and RHOA. Base editing of PIK3CD and PIK3R1 variants in T cells with an engineered T cell receptor specific against a melanoma epitope or in different generations of CD19 chimeric antigen receptor T (CAR-T) cells demonstrates that discovered GOF variants, but not LOF or silent mutation controls, enhanced signaling, cytokine production and lysis of cognate melanoma and leukemia cell models, respectively. Additionally, we show that generations of CD19 CAR-T cells engineered with PIK3CD GOF mutations demonstrate enhanced antigen-specific signaling, cytokine production, and leukemia cell killing, including when benchmarked against other recent strategies.
Absstract of: WO2025101837A1
Described herein are biological devices and extracts useful for detection of different types of cancer. The biological devices include microbial cells transformed with a DNA construct containing genes for producing peroxidase, terminal deoxynucleotidyl transferase (TdT), CA 125, prostatic acid phosphatase, and CA 15-3. Methods for using the devices to detect various cancers, including leukemia, breast cancer, ovarian cancer, and prostate cancer, are also provided herein. In one aspect, the detection methods can make use of commercial ATP detection kits, electromagnetic measurements, or yellow photonic fluorescence measurements.
Absstract of: WO2025098432A1
Disclosed is an mIDH1/NAMPT dual-target inhibitor, which is selected from a compound having a structure as shown in formula (I) or formula (II), or a pharmaceutically acceptable salt, racemate, stereoisomer, prodrug, or solvent compound thereof. The dual-target inhibitor of the present invention can effectively overcome the shortcomings of a single mIDH1 inhibitor and single NAMPT inhibitor having poor efficacy against solid tumors. Said inhibitor can inhibit the malignant proliferation of tumors, has a good therapeutic effect, has low toxicity, and can penetrate the blood-brain barrier, and it is not easy to acquire drug resistance to said inhibitor. Moreover, the inhibitor can be used to prepare a medicament for treating cancer or tumor-related diseases, the cancer or tumor-related diseases comprising multiple myeloma, leukemia, breast cancer, prostate cancer, lung cancer, liver cancer, gastric cancer, bone cancer, brain cancer, head and neck cancer, intestinal cancer, pancreatic cancer, bladder cancer, testicular cancer, ovarian cancer, and endometrial cancer.
Absstract of: WO2025097336A1
The present invention provides an anti-CD30 antigen VHH nanobody. The antibody has the advantages of small relative molecular mass, high affinity, high specificity and stability, good solubility, low immunogenicity and the like. The present invention also constructs a CD30 chimeric antigen receptor and CAR-T cells expressing the chimeric antigen receptor. The use of the CAR-T cells of the present invention for cellular immunotherapy can have a certain treatment effect on various lymphomas.
Absstract of: US2025154599A1
The invention relates to new nucleophosmin protein (NPM) mutants, corresponding gene sequences and relative uses thereof for diagnosis, monitoring of minimal residual disease, prognostic evaluation and therapy of acute myeloid leukaemia (AML).
Absstract of: US2025154268A1
Disclosed in the present invention is an antibody targeting CD25 or a variant thereof. A monoclonal antibody targeting CD25 is a naturally occurring antibody, has an activity of binding to human CD25 and cynomolgus monkey CD25, and has a stronger ADCC effect after being modified with Fc. In-vitro experiments show that the antibody has the effect of clearing Treg and lymphoma cells without blocking an IL-2 signaling pathway.
Absstract of: WO2025099451A1
The present invention relates to compounds of Formula (I), and sub-formulae thereof, that may act as inhibitors of YTHDC1. The invention also relates to pharmaceutical compositions comprising those compounds, and to their use in the treatment of disease and conditions susceptible to YTHDC1 inhibition, such as cancer and in particular acute myeloid leukaemia.
Absstract of: US2025154144A1
Some embodiments of the invention include inventive compounds (e.g., compounds of Formula (I), (II), or (III)) and compositions (e.g., pharmaceutical compositions) which inhibit IRAK and/or FLT3 and which can be used for treating, for example, certain diseases. Some embodiments include methods of using the inventive compound (e.g., in compositions or in pharmaceutical compositions) for administering and treating (e.g., diseases such as hematopoietic cancers, myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), etc.). Additional embodiments provide disease treatment using combinations of the inventive IRAK and/or FLT3 inhibiting compounds with other therapies, such as cancer therapies.
Absstract of: US2025154243A1
Provided herein are methods for treating a subject having chronic myelomonocytic leukemia (CMML), the method comprising: (a) identifying a RAS pathway mutation in tumor cells of the subject, wherein the RAS pathway mutation is a NRAS, KRAS, PTPN-11 and/or CBL mutation; (b) identifying a dominant CBL mutation of CBL variant allele frequency of from <5% to >10%; and (c) administering to the subject identified in step (a) a therapeutically effective amount of an anti-hGM-CSF antibody. Also provided herein are methods for treating a subject having chronic myelomonocytic leukemia (CMML), the method comprising: (a) identifying a RAS pathway mutation in tumor cells of the subject, wherein the RAS pathway mutation is a NRAS, KRAS, PTPN-11 and/or CBL mutation; (b) identifying a dominant CBL mutation of CBL variant allele frequency of from <5% to >10%; and (c) administering to the subject identified in step (a) a therapeutically effective amount of an anti-hGM-CSF antibody lenzilumab and a therapeutically effective amount of a second therapeutic agent. The subject may have a RAS pathway mutation or a RAS pathway mutation and at least one TET2 mutation identified in the tumor cells, an increased percentage of CD116 and CD131 in CD34+ stem and progenitor cells in the subject compared to a healthy subject and/or an increased percentage of CD14+ cells in the subject compared to a healthy subject. A therapeutically effective amount of a hypomethylating agent or hydroxyurea may b
Absstract of: US2025152707A1
The present invention provides combinations and methods using same for the treatment of malignancy, particularly a myeloid malignancy such as acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), myeloproliferative neoplasms (MPN), chronic myeloid leukemia (CML), and chronic myelomonocytic leukemia (CMML). The combination may comprise an antibody or antigen-binding fragment thereof that binds to CD70, and an inhibitor of BCL-2. In certain embodiments, the antibody is ARGX-110 (cusatuzumab). In certain embodiments, the BCL-2 inhibitor is venetoclax. In certain embodiments, the combination provides synergistic treatment of AML. The combination may additionally comprise at least one additional anti-cancer agent.
Nº publicación: WO2025099437A1 15/05/2025
Applicant:
THE INSTITUTE OF CANCER RES ROYAL CANCER HOSPITAL [GB]
FUNDACIO DE RECERCA CLINIC BARCELONA INST DINVESTIGACIONS BIOMEDIQUES AUGUST PI I SUNYER [ES]
INST CATALANA DE RECERCA I ESTUDIS AVANCATS [ES]
THE INSTITUTE OF CANCER RESEARCH: ROYAL CANCER HOSPITAL,
FUNDACI\u00D3 DE RECERCA CL\u00CDNIC BARCELONA-INSTITUT D'INVESTIGACIONS BIOM\u00C8DIQUES AUGUST PI I SUNYER,
INSTITUCI\u00D3 CATALANA DE RECERCA I ESTUDIS AVAN\u00C7ATS
Absstract of: WO2025099437A1
A method of predicting the risk of progression of a cancer of a subject is described. The method can be used to infer the historical growth dynamics of an individual's cancer using fluctuating methylation clocks (FMCs). In the case of, for example, chronic lymphocytic leukaemia (CLL) (at least), the inferred growth rate holds valuable prognostic information, enabling patient stratification beyond that of standard clinical practice.